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An observational study to investigate the relationship between plasma glucosylsphingosine (lyso-Gb1) concentration and treatment outcomes of patients with Gaucher disease in Japan

BACKGROUND: Gaucher disease (GD) is an autosomal recessive disease caused by GBA1 mutations resulting in glucosylceramide accumulation in macrophages. GD is characterized by hepatosplenomegaly, anemia, thrombocytopenia, bone complications, and neurological complications. Glucosylsphingosine (lyso-Gb...

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Detalles Bibliográficos
Autores principales:	Ida, Hiroyuki, Watanabe, Yuko, Sagara, Rieko, Inoue, Yoichi, Fernandez, Jovelle
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9635088/ https://www.ncbi.nlm.nih.gov/pubmed/36329499 http://dx.doi.org/10.1186/s13023-022-02549-6

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